Search clinical trials for: Lysosomal storage disorder
Total 148886 results
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Idorsia Pharmaceuticals Ltd.Active, not recruitingFabry DiseaseUnited States, United Kingdom, Norway, Germany, Australia, Canada, Spain, Switzerland, Austria, Belgium, France, Netherlands, Poland
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SanofiCompletedGaucher's DiseaseUnited States
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AVROBIOTerminatedFabry DiseaseUnited States, Australia, Brazil
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SanofiCompletedGaucher's DiseaseUnited States
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Mandos LLCTerminatedNiemann-Pick Disease, Type CCosta Rica
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TakedaCompleted
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JCR Pharmaceuticals Co., Ltd.Enrolling by invitationMucopolysaccharidosis IIUnited States
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Terence FlotteMassachusetts General Hospital; University of Massachusetts, WorcesterActive, not recruitingTay-Sachs Disease | Sandhoff DiseaseUnited States
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Freeline TherapeuticsCompletedLysosomal Storage Diseases | Fabry DiseaseGermany, United Kingdom
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University Children's Hospital, ZurichSanofiCompleted
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Chile, France, Germany, Italy, Japan, Netherlands, Portugal, Spain, Tunisia, Turkey, United Kingdom
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Chiesi ItaliaCompleted
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Orchard TherapeuticsOspedale San RaffaeleActive, not recruitingLysosomal Storage Disease | Metachromatic LeukodystrophyItaly
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IntraBio IncCompletedNiemann-Pick Disease, Type CUnited States, United Kingdom, Germany, Slovakia, Spain
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SanofiCompleted
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Brazil
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REGENXBIO Inc.Active, not recruitingMucopolysaccharidosis Type II (MPS II)United States, Canada
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Resverlogix CorpWithdrawn
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ZevraDenmarkAvailableNiemann-Pick Disease, Type CUnited States
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National and Kapodistrian University of AthensCompletedFabry DiseaseGreece
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Spark TherapeuticsActive, not recruitingLysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase DeficiencyUnited States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom
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agnes chenUniversity of California, Los Angeles; National Institute of Neurological Disorders... and other collaboratorsTerminatedMucopolysaccharidosis I | Cognitive DeclineUnited States
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Azafaros A.G.Active, not recruitingGM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff DiseaseUnited States, Brazil, France, Germany, Italy, United Kingdom
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Genzyme, a Sanofi CompanyCompleted
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Genzyme, a Sanofi CompanyCompletedSphingomyelin LipidosisUnited States, Belgium, Brazil, France, Germany, Italy, United Kingdom
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Hadassah Medical OrganizationCompleted
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ShireCompleted
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University of FloridaLive Like Bella Pediatric Cancer ResearchCompletedCommon Variable Immunodeficiency | Severe Combined Immunodeficiency | Sickle Cell Disease | Hurler Syndrome | Chronic Granulomatous Disease | Thalassemia | Wiskott-Aldrich Syndrome | Adrenoleukodystrophy | X-linked Lymphoproliferative Disease | Diamond Blackfan Anemia | Bone Marrow Failure Syndrome | Hemophagocytic... and other conditionsUnited States
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseAustralia, Netherlands, United Kingdom, Canada, Czechia, Norway, Slovenia
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Denmark, Belgium, United Kingdom, Norway, Italy, Czechia
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IntraBio IncActive, not recruitingNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, Slovakia, Netherlands, Czechia, Switzerland
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Greenwood Genetic CenterCompletedGalactosialidosis | Sialidosis | Fucosidosis | Aspartylglucosaminuria | Alpha Mannosidosis | Beta Mannosidosis | Mucolipidosis II | Mucolipidosis III | Schindler DiseaseUnited States
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Spain, United Kingdom, Paraguay
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Netherlands, Hungary, United Kingdom, Czechia, Norway, Slovenia, Spain, Finland, France, Italy, Switzerland
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ProtalixChiesi Farmaceutici S.p.A.CompletedFabry DiseaseUnited States, Serbia, Spain, United Kingdom, Australia, Paraguay
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Amicus TherapeuticsCompletedPompe Disease (Late-onset)United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more
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CENTOGENE GmbH RostockActive, not recruiting
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Manchester University NHS Foundation TrustCompletedMucopolysaccharidosis Type IIUnited Kingdom
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AVROBIOTerminatedFabry DiseaseAustralia, Brazil
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Mandos LLCTerminatedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
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AVROBIOTerminatedType 1 Gaucher DiseaseUnited States, Canada
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Homology Medicines, IncWithdrawnMucopolysaccharidosis IIUnited States, Canada
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SanofiCompletedSphingomyelin LipidosisItaly, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain
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University Hospital, BordeauxCompletedChronic Pain | Fabry's DiseaseFrance
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AVROBIOWithdrawn
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ShireTakeda Development Center Americas, Inc.Active, not recruitingMetachromatic Leukodystrophy (MLD)Denmark, Germany, Japan, Australia, France, Brazil, Czechia
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TakedaTakeda Development Center Americas, Inc.Active, not recruitingHunter SyndromeUnited States, Germany, Malaysia, Philippines, Thailand, Dominican Republic, Serbia, Vietnam
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JCR Pharmaceuticals Co., Ltd.Active, not recruitingMucopolysaccharidosis IIBrazil
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National Organization for Rare DisordersFood and Drug Administration (FDA)Active, not recruitingMetachromatic LeukodystrophyUnited States
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LYSOGENETerminatedGM1 GangliosidosisUnited Kingdom, United States, France